Free Paper Focus: Real-world data confirms promise of ocular gene therapy
The latest data from the ongoing PERCEIVE study seems to be consistent with the findings of clinical trials of voretigene neparvovec (VN), the first approved ocular gene therapy for patients with RPE65-mediated inherited retinal dystrophy and sufficient viable retinal cells. “Based on this year 2 interim analysis, the PERCEIVE study demonstrates the safety and effectiveness …
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